Stathmin-2 is new player in the ALS gene-verse
A multi-institutional research team has suggested that aberrant TDP-43 processing of the pre-mRNA of a microtubule-associated protein, stathmin-2 (STMN2), may be the primary contributor to amyotrophic lateral sclerosis (ALS).

New transthyretin aggregation inhibitor outperforms tolcapone
Preveceutical Medical to ramp up preclinical diabetes and obesity dual gene therapy program
Oryzon Genomics awarded grant to support preclinical development of ORY-4001 in ALS
Longboard’s 5-HT2C superagonist yields positive results in preclinical models of epilepsy

Stathmin-2 is new player in the ALS gene-verse
A multi-institutional research team has suggested that aberrant TDP-43 processing of the pre-mRNA of a microtubule-associated protein, stathmin-2 (STMN2), may be the primary contributor to amyotrophic lateral sclerosis (ALS).
New transthyretin aggregation inhibitor outperforms tolcapone
The misassembly of transthyretin (TTR) into toxic amyloid aggregates leads to a variety of degenerative disorders known as TTR amyloidosis (ATTR). Researchers from the Universitat Autònoma de Barcelona and collaborators recently reported on the design and preclinical characterization of PITB, a TTR-selective kinetic stabilizer with potential for the treatment of ATTR.
Preveceutical Medical to ramp up preclinical diabetes and obesity dual gene therapy program
Preveceutical Medical Inc. is working to accelerate the completion of its preclinical diabetes and obesity dual gene therapy program. The company is pursuing a dual-gene therapy strategy using in vitro validated Smart-siRNA sequences, which when paired with its proprietary bioresponsive lipid-nanoparticulate delivery systems, effectively target the PTPN1 gene.
Oryzon Genomics awarded grant to support preclinical development of ORY-4001 in ALS
Oryzon Genomics SA has been awarded a $498,690 grant by the ALS Association to support the regulatory preclinical development of ORY-4001, a highly selective histone deacetylase 6 (HDAC6) inhibitor, for amyotrophic lateral sclerosis (ALS).
Longboard’s 5-HT2C superagonist yields positive results in preclinical models of epilepsy
Serotonin 5-HT2 receptor agonists have shown efficacy in treating seizure disorders, such as epilepsy. At the American Epilepsy Society meeting this week, Longboard Pharmaceuticals Inc. presented data on their 5-HT2C receptor superagonist LP-352 for the treatment of epilepsy.
Holiday notice
In accordance with the publishing schedule, BioWorld Science will not be published on Wednesday, Dec. 6, 2023.
NLS Pharmaceutics enters option agreement with Aexon Labs for dual orexin receptor agonist platform
NLS Pharmaceutics Ltd. has entered into an exclusive worldwide option agreement with Aexon Labs Inc.
GT Biopharma submits IND application for GTB-3650 for CD33+ leukemia
GT Biopharma Inc. has submitted an IND application to the FDA for the development of GTB-3650 for the treatment of patients with CD33+ leukemia.
Seismic Therapeutic closes series B financing to advance immunology pipeline
Seismic Therapeutic Inc. has closed a $121 million series B financing to support progression of the company’s two lead programs through clinical proof of mechanism.
Prelude Therapeutics patents SMARCA2 and SMARCA4 protein degraders for treatment of cancer
Research at Prelude Therapeutics Inc. has led to the development of SMARCA2 and/or SMARCA4 degradation inducers.

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Conferences

Favorable preclinical profile of investigational gene therapy for TLE reported
Gene therapy
Temporal lobe epilepsy (TLE) is the most common form of focal epilepsy characterized by recurrent seizures originating in the hippocampus and with sprouting of mossy fiber axons that contribute to new recurrent synaptic excitability in the dentate gyrus (DG).
Iama Therapeutics’ IAMA-6 shows promise in preclinical mesial temporal lobe epilepsy
Neurology/psychiatric
Intracellular chloride levels are crucial for physiological brain development and correct functioning. On the other hand, aberrant expression of chloride importer NKCC1 (basolateral Na-K-Cl symporter, SLC12A2) and exporter KCC2 (K-Cl cotransporter 2, SLC12A5) is involved in neurodevelopmental and...
Bifunctional molecule capsid modulator and cGAS agonist achieves complete cure in AAV-HBV mice
American Association for the Study of Liver Diseases
LW-231, under development by Shanghai Longwood Biopharmaceuticals Co. Ltd., is a novel bifunctional molecule designed to simultaneously modulate hepatitis B virus (HBV) capsid assembly and activate cGAS-STING pathway. Preclinical data were recently presented.
Stem, immune and nervous cells all forces for tumor progression
Cancer
One of the difficulties for preventing the evolution of a tumor is that cancer progression can be promoted by undifferentiated or migrating cells whose states could follow different directions. At the 40th edition of Barcelona Biomed conferences at the Institute for Research in Biomedicine (IRB...

Cancer

Discovery of dual-target inhibitors of microtubule polymerization and JAK2
Synthesis and optimization of substituted 2-amino[1,2,4]-triazolopyrimidines and related heterocycles by Wuyi University investigators has led to the identification of compound [I], and its water-soluble phosphate sodium salt prodrug, compound [II].

Infection

Ebviously presents in vitro data on EBV vaccine candidate
Immune
Ebviously GmbH, a spin-off from Helmholtz Munich (HMGU), has presented new in vitro data for its Epstein-Barr virus (EBV) vaccine candidate EBV-001. Based on noninfectious virus-like particles (VLPs) derived from EBV, EBV-001 is designed as a highly immunogenic, multiantigen vaccine to prevent...

Dual Mcl-1 and Bcl-2 inhibition as promising approach for treating tuberculosis
Tuberculosis still kills a lot of people worldwide (1.6 million deaths per year). Previous findings demonstrated that induced myeloid leukemia cell differentiation protein Mcl-1 inhibitors reduced the growth of Mycobacterium tuberculosis in human macrophages, but when inhibiting multiple proteins...
New dual inhibitors of host proteases TMPRSS2 and CTSL/CTSB as potential anti-SARS-CoV-2 agents
Coronavirus
Current antiviral agents for COVID-19 treatment target viral proteins, which are susceptible to mutation during SARS-CoV-2 evolution. A potential strategy to fight emerging drug resistances is the development of compounds targeting host proteins that are indispensable for the viral life cycle.

Neurology/psychiatric

Psychogenics licenses rights to ENT1 inhibitor for neuropsychiatric disorders
License
PGI Drug Discovery LLC, the drug discovery arm of Psychogenics Inc., has entered into an exclusive license agreement with F. Hoffmann-La Roche Ltd. to obtain global development, manufacturing and commercialization rights to RO-7117997. Psychogenics intends to evaluate RO-7117997 as a potential...
Nurexone reports new siRNA sequences with potential for spinal cord injury
RNA
Nurexone Biologic Inc. has reported results from laboratory tests of its secondary two proprietary sequences, showing promise for the treatment of spinal cord injuries.
Cytokinetics describes new bicyclic piperazinones to modulate skeletal muscle contractility
Patents
Cytokinetics Inc. has identified bicyclic piperazinones with the ability to modulate skeletal muscle contractility reported to be useful for the treatment of amyotrophic lateral sclerosis, spinal muscular atrophy, myasthenia gravis, chronic obstructive pulmonary disease, stress urinary...
Roche divulges new GABRA2 PAMs for medical imaging of neurological disorders
Diagnostics
Researchers at F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have synthesized radiolabeled compounds acting as GABA(A) receptor subunit α2β2γ1 (GABRA2) positive allosteric modulators acting as positron-emission tomography (PET) and autoradiography imaging agents reported to be useful for the...
Brain penetration profile of Nodthera’s NLRP3 inflammasome inhibitor described
Inflammatory
Aberrant NLRP3 inflammasome activation is at the root of a wide number of conditions, from heart, gastrointestinal, kidney or liver disorders to neuroinflammatory diseases such as Parkinson’s disease or multiple sclerosis. Researchers from Nodthera Ltd. have reported on NT-0796, an NLRP3...

Endocrine/metabolic

FDA clearance for first-in-human study of Uniqure’s AMT-191 for Fabry disease
Regulatory
Uniqure NV has received FDA clearance of its IND application for AMT-191, the company’s gene therapy candidate for Fabry disease.

Biomarkers

miR-92a confers protection against inflammation-driven neurodegeneration
Neurology/psychiatric
MicroRNAs (miRNAs) play an important role in modulating neuronal stress responses, but their impact on neuroprotection has been poorly studied. Multiple sclerosis (MS) is the most common inflammatory disease affecting the central nervous system. Researchers have recently attempted to identify...

Somatic Ras-MAPK activating variants linked to mesial temporal lobe epilepsy
Conferences
Researchers from The Brigham and Women's Hospital presented data from a study that aimed to evaluate the role of somatic variants in drug-resistant mesial temporal lobe epilepsy (MTLE). High-coverage whole-exome sequencing was conducted using DNA samples derived from the hippocampus and paired...
Researchers find de novo CAMK2B variant in patient with tetralogy of Fallot
Conferences
CAMK2B encodes calcium/calmodulin-dependent protein kinase type II subunit β, a kinase expressed in the brain that has an important role in synaptic plasticity. Genetic variations are associated with neurodevelopmental disorders.

Gastrointestinal

Esperion Therapeutics discovers new ACLY inhibitors
Patents
Esperion Therapeutics Inc. has described ATP citrate lyase (ACLY) inhibitors reported to be useful for the treatment of cancer and more.
ZHX2 promotes liver recovery after acute liver injury
Dysfunction in the mitochondria contributes to the development of acute liver injury (ALI). There is emerging evidence indicating the mitochondria is key to maintain liver homeostasis and survival; thus, controlling its functioning is important...
Novel hDHODH inhibitor for inflammatory bowel disease described
Human dihydroorotate dehydrogenase (hDHODH) is a mitochondrial enzyme participating in de novo pyrimidine biosynthesis essential for T- and B-lymphocyte proliferation.
Investigators find lactic acid bacteria strain that alleviates IBS in rats
Irritable bowel syndrome (IBS) is a functional gastrointestinal disorder where visceral hypersensitivity (VH), which causes discomfort and negatively impacts the quality of life, is influenced by stressful events. In recent years, the use of...
Lerna Biopharma presents first-in-class GalNAc-siRNA therapeutic for treatment of liver diseases
Conferences
Researchers from Lerna Biopharma Pte. Ltd. (formerly Cargene Therapeutics Pte. Ltd.) recently presented the discovery and preclinical evaluation of a first-in-class GalNAc-siRNA therapeutic, CG-LR1, being developed for the treatment of liver...
CVI Pharmaceuticals CVI-2742 shows promise for treating NASH
Conferences
CVI Pharmaceuticals has presented preclinical data on their thyroid hormone receptor beta (THRB) selective agonist CVI-2742 for the potential treatment of NASH. Selective activation of the THRB contributes to ameliorating the symptoms of...

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